The Societal Cost of Duchenne Muscular Dystrophy in Brazil: A Burden of Illness Study

Speaker(s)

Grossklauss LF1, Sobrinho JCLB2, Fernandes AR3, Ferreira PK4, Barbosa RDCGDA5, Zanotelli E6, Costa M7, Gurgel-Giannetti J8, Ung B9, Iff J9, Evans J10
1TDN/Afip Tratamento de Doenças Neuromusculares/Associação Fundo de Incentivo a Pesquisa, São Paulo, Brazil, 2Associação Baiana de Distrofias Muscular (ABDM), Sao Paulo, Brazil, 3Universidade Federal Fluminense, Fluminense, Brazil, 4Faculdade Pequeno Príncipe, Curitiba, Brazil, 5Programa VENTLAR da FHEMIG - Fundação Hospitalar do Estado de Minas Gerais, Gerais, Brazil, 6Faculdade de Medicina da Universidade de São Paulo (FMUSP), São Paulo, Brazil, 7Escola Bahiana de Medicina e Saúde Pública, Salvador, Brazil, 8Hospital das Clínicas da UFMG, Belo Horizonte, Brazil, 9Sarepta Therapeutics Inc., Cambridge, MA, USA, 10Prime HCD, Knutsford, CHE, UK

Presentation Documents

OBJECTIVES: A burden-of-illness (BOI) study was conducted to quantify societal costs of Duchenne muscular dystrophy (DMD) in Brazil, including direct medical, direct nonmedical, and indirect costs.

METHODS: Physicians completed an electronic case report form that captured demographic, clinical, and hospital health-resource use within the past 12 months. A patient and caregiver survey captured direct nonmedical and indirect costs. Male pediatric (>4 years) and adult patients diagnosed with DMD for ≥12 months were recruited by DMD specialists across 10 sites. Patients were stratified by health state.

RESULTS: Among 130 patients, mean age was 13.4 (±4.6) years; mean age at diagnosis was 6.2 (±2.6) years. Patient distribution across health states was early ambulatory (EA; 18%); late ambulatory (LA; 12%); early nonambulatory (ENA; 57%); and late nonambulatory (LNA; 13%). Mean direct medical costs ranged from a low of R$7,935 (±1,172) in EA to a high of R$12,922 (±8,429) in LNA. Across health states, most direct medical costs were attributed to medical device use (83%), with wheelchair purchase in the past 12 months rising to 82% in ENA. Mean direct nonmedical costs were much higher than direct medical costs, ranging from R$24,626 (±28,564) in EA to R$84,724 (±57,326) in LNA, driven by informal caregiving (61%). Mean indirect costs, mostly consisting of costs related to the caregiver discontinuing work (69%), ranged from R$16,247 (±12,557) in LA to R$18,750 (±14,920) in ENA. Mean total societal costs increased across health states: EA: R$50,656 (±29,728); LA: R$64,368 (±45,416); ENA: R$96,741 (±58,913); LNA: R$114,853 (±68,625), with direct medical, direct nonmedical, and indirect costs contributing 11%, 74%, and 15%, respectively, for LNA.

CONCLUSIONS: These findings from the largest DMD BOI study in Brazil to date demonstrate the significant impact of DMD on societal costs from a patient and caregiver perspective, which increase as patients progress through the course of disease.

Code

EE618

Topic

Economic Evaluation

Disease

No Additional Disease & Conditions/Specialized Treatment Areas, Rare & Orphan Diseases